Scientists corrected the defective genes of Duchenne muscular dystrophy disease by genome editing
Duchenne muscular dystrophy (DMD) is a devastating progressive disease that usually starts in early childhood. DMD occurs because of a mutated gene which fails to produce dystrophin, a protein which is important for the normal structure and function of muscle. Currently, no definitive treatments are available except for palliative therapy, which can only delay the symptoms of the disease. Moreover, available treatments are not completely effective, as they treat just one aspect of the disease, and they may have side effects in the long run.